We are his family: the dad Cristian, the mother Ilaria and his 13 years old sister Serena. Two years ago, when Diego was diagnosed with the disease, our world literally shattered in front of our eyes. To find out about our baby disease was a terrible experience in which the initial disbelief was followed by anxiety and fear. Accept the prognosis was not easy, but we reacted and began to get informed about what was going on in Diego’s body. We began to look for more effective therapies. In Italy we have desperately tried all the available treatments and all the possible alternatives resources. Unfortunately none of this was enough to stop the degeneration of the disease. Yet, we could not surrender, we haven’t given up, we didn’t stop. We contacted several associations of parents with children with rare diseases, to find out if someone had similar conditions as Diego. We were looking for news and hope for a possible cure that could have some sort of positive results. We could not find the right answers, but we kept asking, chasing hope, starting to search internationally as well. We then managed to find a drug being tested in the United States. The preliminary results on the drug testing seemed very encouraging, but the United States were so far away… The contacts with the pharmaceutical company were long and complicated, we encountered many obstacles. We had to figure out what kind of cure it was, how to access to it and, not least, to solve the problems related to Visa/Immigration, traveling, etc. After a troubled path, we have been granted a Visa for stay temporarily in the US on condition of not working while in the Country. We finally managed to be included in the trial that started in August in Houston, Texas.